Maintaining GALNS Enzyme Activity for Extended Periods

SLU ID 17-008 | Delivering GALNS Enzyme Using an Injectable Hydrogel Depot

Background

GALNS is a gene that provides instructions for producing the N-acetylgalactosamine 6-sulfatase enzyme, which is located in compartments within cells, called lysosomes, which break down and recycle different types of molecules. This enzyme specifically removes a sulfate from a glycosaminoglycan (GAG) called keratan sulfate, which is abundant in cartilage and the cornea. Mucopolysaccharidosis type IV (MPS IV), also called Morquio syndrome, is a progressive condition that mainly affects the skeleton and is known to be caused by more than 148 different mutations in the GALNS gene. There are many efforts to develop GALNS-based enzyme replacement therapies. However, the GALNS enzyme loses complete activity in less than 24 hours when in solution at 37 degrees Celsius.

Overview

Researchers at Saint Louis University have developed an injectable polyethylene glycol (PEG) hydrogel depot to deliver the GALNS enzyme and maintain enzyme activity for up to 7 days.

Benefits

The potential benefits of this technology include:

• Increasing the effectiveness of GALNS treatments

• Minimizing the frequency of GALNS infusions for patients

• Minimizing the dosage requirements

• Minimizing patient hospitalization time

• Minimizing treatment costs

Applications

• This technology has potential application for treating genetic diseases caused by mutations in the GALNS gene.

Opportunity

Saint Louis University is seeking a partner to further develop and commercialize this technology.